University of Pennsylvania Cancer Center
Last Modified: May 19, 1998
Antisense therapy is a novel cancer treatment approach that works at the genetic level to interrupt the process by which cancerous cells produce certain proteins. An antisense compound consists of a single strand of DNA designed in the laboratory to complement the single strand of messenger RNA (mRNA) produced by the cancer cell. These two stands fuse, which blocks transcription of the protein.
One of these cancer-causing proteins, known as "Raf," plays an active role in the development of some solid tumors. This Phase I trial tested the safety of "Raf-IK antisense ODN" on 31 patients. This agent is designed to identify and target solid tumors with Raf mutations, and to stop production of the protein, thereby preventing additional proliferation of genetically-mutated cancer cells. The study confirmed that the Raf gene could be suppressed, and while the research was designed only to test safety and the viability of the approach, two of the patients in the study -- one with colorectal cancer and one with renal cancer, showed no signs of cancer progression.